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BioWorld - Sunday, May 28, 2023
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  • Endosome

    Experimental painkiller blocks GPCR signaling inside the cell

    Researchers have developed a new highly effective therapeutic for pain relief by altering the chemical properties of an antinausea drug, netupitant. The modified drug is able to enter the intracellular membrane-bound endosome and target the GPCRs therein, rather than at the cell surface, that leads to optimal pain relief. The study, published in the Proceedings of the National Academy of Sciences (PNAS) on May 22, 2023, was led by Nigel Bunnett, Professor and Chair of Molecular Pathobiology at NYU College of Dentistry, and illustrates how GPCR-mediated pain signaling occurs inside the endosomes rather than at the surface, highlighting the need for drugs that can reach receptors within the cells itself.
  • Inhibition of YTHDF2 results in a more effective and safe radiotherapy

    In a study published in Cancer Cell on May 25, 2023, researchers from the University of Chicago and colleagues reported that the inhibition of YTHDF2, an immune suppressor protein, can be a valuable strategy to improve radiotherapy outcomes by overcoming resistance while enabling extra help from the immune system.
  • Experimental painkiller blocks GPCR signaling inside the cell

    Researchers have developed a new highly effective therapeutic for pain relief by altering the chemical properties of an antinausea drug, netupitant. The modified drug is able to enter the intracellular membrane-bound endosome and target the GPCRs therein, rather than at the cell surface, that leads to optimal pain relief. The study, published in the Proceedings of the National Academy of Sciences (PNAS) on May 22, 2023, was led by Nigel Bunnett, Professor and Chair of Molecular Pathobiology at NYU College of Dentistry, and illustrates how GPCR-mediated pain signaling occurs inside the endosomes rather than at the surface, highlighting the need for drugs that can reach receptors within the cells itself.
  • Inhibition of YTHDF2 results in a more effective and safe radiotherapy

    In a study published in Cancer Cell on May 25, 2023, researchers from the University of Chicago and colleagues reported that the inhibition of YTHDF2, an immune suppressor protein, can be a valuable strategy to improve radiotherapy outcomes by overcoming resistance while enabling extra help from the immune system.
  • Other news to note for May 26, 2023

    Additional early-stage research and drug discovery news in brief.
  • PAK4 inhibitor from Hyperway Pharmaceutical has improved safety and PK profile

    Work at Hyperway Pharmaceutical Co. Ltd. has led to the discovery of a novel PAK4 inhibitor...
  • Hanall and Daewoong partner with Nurron to advance compounds for neurodegenerative disorders

    Hanall Biopharma Co. Ltd. and Daewoong Pharmaceutical Co. Ltd. have entered into...
  • Bright Angel Therapeutics patent describes new Hsp90 inhibitors

    Bright Angel Therapeutics Inc. has patented heat shock protein 90 inhibitors reported to be useful...
  • Psilera nominates non-hallucinogenic psilocybin derivative as lead clinical candidate

    Psilera Inc. has selected a novel, non-hallucinogenic psilocybin derivative, PSIL-006...
  • MyoDys45-55 restores dystrophin and improves functional measures in murine model of DMD

    Myogene Bio LLC has developed an approach through CRISPR7Cas9 technology...
  • Teon Therapeutics identifies new cannabinoid CB2 receptor modulators for cancer

    Teon Therapeutics Inc. has reported 4-hydroxy-2-oxo-1,2-dihydro-1,8-naphthyridine-3-carboxamide...
  • LTB4 antagonist shows efficacy in rat inflammation model

    Art concept for inflamed human tissue
  • Humanwell Healthcare patents new 15-PGDH inhibitors

  • Series D financing at Benchsci to expand Ascend AI drug discovery platform

  • Evaxion develops genetic adjuvant technology to boost immune response to viral, bacterial and cancer vaccines

    DNA in test tubes
  • Fochon Biosciences describes new PI3Kα inhibitors for cancer

  • AAV9-CLN5 improves symptoms in mice with Batten disease

  • Researchers create new mouse model of DMD using knock-in of human exon 50

    Muscular dystrophy
  • Betta Pharmaceuticals prepares and tests new HIF-2α inhibitors

BioWorld Insider Podcast

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Conferences

  • Degradation of motor neurons

    Next-generation gene therapy shows superior efficacy for spinal muscular atrophy

    Neurology/Psychiatric
    Spinal muscular atrophy (SMA) is caused by mutations in the SMN1 gene, which encodes survival motor neuron 1, leading to reduced protein expression levels and degeneration of motor neurons in the spinal cord, with the consequent muscle atrophy. There is thus a need for new AAV gene therapies for...
  • Illustration of the inside of an eye with macular degeneration

    SKG-0106 shows durable efficacy in preclinical models of neovascular AMD

    Ocular
    Researchers from Skyline Therapeutics (Shanghai) Co. Ltd. presented preclinical data for the new recombinant AAV vector therapeutic SKG-0106, being developed for the treatment of neovascular (wet) age-related macular degeneration (AMD).
  • Alpha-galactosidase enzyme

    AAV9-GLA restores α-galactosidase levels in murine model of Fabry disease

    Endocrine/Metabolic
    Fabry disease is a metabolic disease characterized by a deficiency in the lysosomal α-galactosidase enzyme caused by mutations in the GLA gene. This leads to substrate accumulation in the lysosomes, cellular dysfunction and organ damage.
  • Illustration of intestines overlayed on human torso

    NTX-1175 channel blocker is effective in visceral pain models

    Gastrointestinal
    Patients with irritable bowel syndrome (IBS) have chronic abdominal pain as a result of visceral hypersensitivity. Voltage-gated sodium channels control cellular excitability and are involved in the pathophysiology of several functional gastrointestinal disorders. Researchers from Nocion...
More in Conferences

Today's news in brief

  • Other news to note for May 26, 2023

  • Respiratory

  • Dermatologic

  • Cardiovascular

  • Hematologic

  • Nephrology

Patents

  • Simcere Zaiming Pharmaceutical reports new CBL-B inhibitors for cancer and autoimmune disease

  • China Pharmaceutical University and Jiangsu Chia Tai Tianqing Pharmaceutical identify Wee1 inhibitors for treatment of cancer

  • Kymera Therapeutics prepares and tests TYK2 degradation inducers

  • Perha Pharmaceuticals patents CLK1, CLK4 and/or DYRK1A inhibitors

  • Marvel Biotechnology patent details new compounds for treatment of depression

  • Complement factor B inhibitors reported in Hansoh patent

  • Reunion Neuroscience identifies 5-HT2A receptor agonists

  • Roche prepares and tests new LpxH inhibitors for the treatment of bacterial infections

  • Twentyeight-Seven describes new CLK3 inhibitors

  • Cancer Research Technology and Merck KGaA patent YAP1/TEAD interaction inhibitors

Cancer

  • Cancer cells under magnifying glass

    Discovery of new IDO-1 inhibitors with potent anticancer activity

    Researchers from Guilin Medical University (GLMU) reported the discovery and preclinical evaluation of novel indoleamine 2,3-dioxygenase 1 (IDO-1) inhibitors as potential anticancer agents. Synthesis and optimization of a series of chromone-oxime derivatives containing piperazine sulfonamide...
  • Bioray receives IND clearance in China for BRY-812 ADC targeting LIV-1

    Antibody-drug conjugate
  • Antengene's Claudin 18.2 ADC ATG-022 awarded US orphan drug designations for gastric and pancreatic cancer

    Antibody-drug conjugate
  • Allosteric TKI HS-10382 shows efficacy in several CML models

    Conferences
  • Idorsia Pharmaceuticals prepares and tests new CCR6 antagonists

    Patents
More in Cancer
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Infection

  • Colorized transmission electron micrograph of rabies virus.

    AAV9-based therapy neutralizes rabies viral infection in neurons

    Conferences
    Rabies virus is among the most neurotrophic known virus and it has a fatality rate of almost 100%. There is a need for antibody-induced protection in the central nervous system, since rabies virus can cross the blood-brain barrier but antibodies cannot. Investigators from Auburn University and the...
  • Westvac announces NMPA clearance for clinical trials of bivalent and trivalent COVID-19 protein vaccines

    Immune
    China's National Medical Products Administration (NMPA) has given drug clinical trial approval for two new COVID-19 vaccines against the current XBB variants developed by Westvac Biopharma Co. Ltd. and West China Medical Center at Sichuan University.
  • Assembly Biosciences patents new compounds for hepatitis B

    Patents
    Assembly Biosciences Inc. has disclosed heteroaryl carboxamide derivatives reported to be useful...
  • Immunic presents XAVI-101, a novel DHODH inhibitor with broad-spectrum antiviral properties

    Conferences
  • Preclinical efficacy of ALG-097558 in SARS-CoV-2 hamster model presented

    Conferences
  • Kimera receives IND approval to study mesenchymal stem cell exosomes to treat COVID-19

    Coronavirus
  • Researchers in Spain discover new antimalarials

    Patents
More in Infection

Neurology/Psychiatric

  • onward.jpg

    Let there be walk: Onward Medical sees first use of movement-restoring lead

    Artificial intelligence
    The researchers who enabled patients with spinal cord injuries to walk independently after implanting programmable electrodes below their lesions have now taken things one step further, restoring direct communication from the brain to the spinal cord, enabling the brain rather than an external...
  • Illustration of prescription pill bottle with DNA on the label.

    Gene expression study gives clues to pain insensitivity syndrome

    Researchers have used cell culture experiments to understand how gene expression was affected in a patient with a rare pain insensitivity syndrome, and have identified a network of hundreds of genes whose expression was changed compared to sex-matched controls. Published online in the journal Brain...
  • Epileptic brain and abnormal EEG wave discharges

    AEG-1 regulates granule cell dispersion, seizure development in model of temporal lobe epilepsy

    Conferences
    Researchers from Kyungpook National University presented data from a study that aimed to investigate the endogenous mechanisms involved in granule cell dispersion (GCD) and its role in epileptic seizures in temporal lobe epilepsy (TLE).
  • UCSFDBS2.jpg

    Where does it hurt? For chronic pain, in the orbitofrontal cortex

    Long-term brain recordings from four patients with chronic pain have led investigators at the University of California at San Francisco to identify brain signals that could serve as biomarkers for each individual patients’ pain. The study, which was published online in Nature Neuroscience on May...
  • Merck Sharp & Dohme reports RIPK1 inhibitors for ALS and inflammatory disorders

    Patents
    Research at Merck Sharp & Dohme Corp. has led to the development of receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis (ALS) and inflammatory disorders.
More in Neurology/Psychiatric

Immune

  • Exo Therapeutics announces TBK1 exosite inhibitor program for autoimmune diseases

  • Monte Rosa announces MRT-6160 as molecular glue degrader of VAV1 for autoimmune diseases

  • Hummingbird Bioscience HMBD-011 shows promise for autoimmune disorders

  • Dualyx completes series A financing to advance Treg therapies for autoimmune diseases

  • Carmot Therapeutics patents USP28 inhibitors

  • Absci and University of Oxford partner on discovery and development of therapies for immune-mediated diseases

  • GPR183 antagonists detailed in Immunophage Biomedical patent

  • COVID vaccine myocarditis is due to inflammation

  • New bivalent exosome vaccine platform may offer potent, broader immunity against SARS-CoV-2

  • Tonix receives IND clearance to study TNX-1500 to prevent organ rejection in kidney transplant patients

Endocrine/Metabolic

  • Bloomsbury Genetic’s liver-targeted AAV-LK03 gene therapy BGT-OTCD cleared to enter clinic

    Gene therapy
    The U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) and the Gene Therapy Advisory Committee (GTAC) have approved a clinical trial application (CTA) submitted by University College London (UCL) to initiate a phase I/II trial of BGT-OTCD, Bloomsbury Genetic Therapies Ltd.’s...
  • Researchers optimize biphenyl scaffold molecules as potent FABP4 inhibitors

    Inflammatory
  • Terns Pharmaceuticals divulges new GLP-1 receptor agonists for diabetes and liver diseases

    Patents
  • Gene therapy to reverse T2D shows proof of concept at ASGCT

    Science
  • Cgenetech discovers new GLP-1 receptor agonists for diabetes

    Patents
More in Endocrine/Metabolic
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Biomarkers

  • Microscopic image of acute myeloid leukemia (AML) cells.

    Low miR-143 expression and high MSI2 expression are associated with poor prognosis in AML

    Cancer
    Researchers from Wenzhou Medical University and affiliated organizations have published data from a study that aimed to investigate the relationship between the tumor suppressor microRNA-143 (miR-143) and RNA-binding protein Musashi homolog 2 (MSI2), the abnormal expression of which has been...
  • SDC4 confers poor survival in gastric cancer

    Cancer
    Gastric cancer persists as the fourth leading cause of cancer-related deaths. Syndecans (SDCs) are a family of four transmembrane heparan sulfate proteoglycans involved in cell proliferation, migration and adhesion, among others, and syndecan-4 (SDC4) expression has been shown to be up-regulated...
  • SNP in miR-146a is a prognostic marker in acute coronary syndrome

    Cardiovascular
    Despite advances in diagnosis and treatment, cardiovascular disease remains the leading cause of death worldwide. Single-nucleotide polymorphisms (SNPs) in microRNAs are known to play important roles in acute coronary syndrome (ACS). MicroRNA 146a (miR-146a) is significantly upregulated in human...
  • Study finds TNFRSF14 and LIGHT levels are elevated in primary sclerosing cholangitis

    Conferences
  • Tear cytokine composition useful to diagnose dry eye disease, researchers find

    Conferences
  • CD138 serum levels are lower in patients with EoE

    Conferences
  • New missense mutation in SLC6A6 associated with Leber congenital amaurosis pathology

    Conferences
More in Biomarkers

Gastrointestinal

  • Small-molecule AXIN stabilizer from University of Maryland inhibits liver fibrosis

    Conferences
    Researchers from the University of Maryland presented preclinical data for YA-6060, a novel small molecule showing dual activities of Wnt inhibition and AMPK activation via the mechanism of AXIN stabilization. Since both Wnt/β-catenin and AMPK...
  • Insilico Medicine identifies new PHD2 inhibitors

    Patents
    An Insilico Medicine IP Ltd. patent describes Egl nine homolog 1 (EGLN1; HPH-2; PHD2) inhibitors reported to be useful for the treatment of ulcerative colitis and Crohn’s disease.
  • LPA1 receptor agonist improves NASH-related outcomes in preclinical models

    Conferences
    Currently, there is no FDA-approved drug for nonalcoholic steatohepatitis (NASH), which has evolved into the second leading cause of liver transplantation in the U.S. Researchers from Children’s Hospital Los Angeles and Epigen Biosciences Inc....
  • Researchers present data from murine studies of PAM-369, a muscarinic M3 receptor PAM for enteropathy

    Conferences
    Researchers from Kyushu University and Mochida Pharmaceuticals Co. Ltd. disclosed recent data along with the chemical structure of PAM-369, a novel muscarinic acetylcholine M3 receptor positive allosteric modulator (PAM), being developed for the...
  • Researchers report discovery of ROR-107, a GPBAR1 agonist and RORγt inverse agonist for IBD

    Conferences
    Inflammatory bowel disease (IBD) is a chronic, immunologically mediated disorder of the gastrointestinal tract in which tissue damage and sustained inflammation lead to long-term dysfunction of the gastrointestinal tract.
  • Tackling spores is new strategy for recurrent Clostridioides difficile

    Infection
    An experimental antibiotic was able to prevent recurrent Clostridioides difficile infections (rCDI) by killing bacterial spores as well as growing spores. Researchers from the University of Notre Dame reported their findings in the Proceedings of...
More in Gastrointestinal

BioWorld Insider Podcast

One-on-one with medical innovators

podcast microphone, sound waves on purple backgroundBreakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.


Recent episodes:

  • Better times ahead for the biopharma sector? Could be, the new numbers say
  • Radiopharmaceuticals: The next big disrupter?
  • Cambrian carves out a new niche as it works to keep people from getting sick
  • Rethinking obesity: Fitness may be more directly linked to health than weight
  • Looking ahead to 2023: CEOs contemplate the new normal
  • Psychedelic evolution: Mindset Pharma looks to change mental health treatment
  • $1B+ biopharma deals keep values afloat, even amid muted volume
  • Extending the human lifespan
  • New therapies vie to change fatal course of amyotrophic lateral sclerosis
  • Biopharma’s correction? 2022 1Q investments are both up and down

View all

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